The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Inherited Diseases

Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR) is a promising innovative technology for genomic editing that offers scientists the chance to edit DNA structures and change gene function. It has several possible uses consisting of editing inherited deficiencies, treating, and reducing the spread of disorders. Recently, reports have demonstrated the creation of synthetic RNA molecules and supplying them alongside Cas9 into genome of eukaryotes, since distinct specific regions of the genome can be manipulated and targeted. The therapeutic potential of CRISPR/Cas9 technology is great, especially in gene therapy, in which a patient-specific mutation is genetically edited, or in the treating of human disorders that are untreatable with traditional treatments. This review focused on numerous, in vivo, in vitro, and ex vivo uses of the CRISPR/Cas9 technology in human inherited diseases, discovering the capability of this versatile in medicine and examining some of the main limitations for its upcoming use in patients. In addition to introducing a brief impression of the biology of the CRISPR/Cas9 scheme and its mechanisms, we presented the utmost recent progress in the uses of CRISPR/Cas9 technology in editing and treating of human genetic diseases.

Keywords: CRISPR/Cas9; gene editing; genetic diseases; rectifying; treatment.

FIGURE 1

CRISPR mechanism. The Guide RNA hybridizes with the target DNA sequence and directs Cas9 endonuclease (colored in yellow) to generate a double-strand break. Subsequently, mutant DNA is generated from the repair process of DNA, through either the homology-directed repair (HDR) or the non-homologous end-joining (NHEJ) mechanism. The final mutant DNA could include deletion or insertion of DNA sequence (NHEJ), or replacement with a particular DNA sequence used as a marker for further study (encoding for a fluorescence protein, tag protein, antibiotics, or the recognition sequence for a restriction enzyme digestion).